A hematologist and oncologist, Mukherjee is also known for his work on the formation of blood and the interactions between the micro-environment (or "niche") and cancer cells. Recently, the Government of India has conferred its fourth highest Civilian Award Padma Shri upon Mukherjee.[5]

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Siddhartha Mukherjee was born in New Delhi, India to Sibeswar Mukherjee, an executive with Mitsubishi, and Chandana Mukherjee, a former schoolteacher from Kolkata. He attended St. Columba's School in Delhi where he won the school's highest award the 'Sword of Honour' in 1988. As a biology major at Stanford University, he worked in Nobel Laureate Paul Berg's laboratory defining cellular genes that change the behaviours of cancer cells.

In 2010, Simon & Schuster published his book, The Emperor of All Maladies: A Biography of Cancer,[9] detailing the evolution of diagnosis and treatment of human cancers from ancient Egypt to the latest developments in chemotherapy and targeted therapy.[10]The Oprah magazine listed it in its "Top 10 Books of 2010".[11] It was also listed in "The 10 Best Books of 2010" by The New York Times[12] and the "Top 10 Nonfiction Books" by Time.[13]

A hematologist and oncologist by training, Mukherjee's scientific work addresses the links between normal stem cells and cancer cells. Mukherjee's lab has performed investigations on the microenvironment—or niche—of stem cells, focusing particularly on blood-forming stem cells. Blood-forming stem cells (called hematopoietic stem cells or HSCs) reside in the bone marrow in very specific microenvironments. Osteoblasts, or bone-forming cells, comprise an important element of this niche, and they regulate the physiology of HSCs, by providing HSCs with signals to divide, remain quiescent, or maintain their stem cell properties. Mukherjee's scientific work has been recognised through many NIH and private foundation grants, including the prestigious NIH "Challenge Grant" awarded to pioneering researchers in 2009.

In work performed with collaborators in the 1990s and 2000s, Mukherjee's lab identified genes and chemicals that can alter the microenvironment, or niche, and thereby alter the behaviour of normal stem cells, as well as cancer cells. Two such chemicals studied in the lab – proteasome inhibitors and Activin A inhibitors—are currently in clinical trials with novel therapeutic uses as defined by these studies. The lab has also identified novel genetic mutations in myelodysplasia and acute myelogenous leukaemia and has played a leading role in finding therapies for these diseases in the clinical setting. Mukherjee's lab is based at Columbia University's Herbert Irving Comprehensive Cancer Center. Previously, he was affiliated with the Harvard Stem Cell Institute and with Massachusetts General Hospital in Boston.